On 21 March 2012, orphan designation (EU/3/12/973) was granted by the European Commission to NDA Regulatory Science Ltd, United Kingdom, for recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome).
The sponsorship was transferred to NDA Group AB, Sweden, in December 2013 and subsequently to Ultragenyx UK Limited, United Kingdom, in May 2015.
Recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome) has been authorised in the EU as Mepsevii since 23 August 2018.
This medicine is now known as vestronidase alfa.
The sponsor’s address was updated in November 2020.
Recombinant human beta-glucuronidase (vestronidase alfa)
Treatment of mucopolysaccharidosis type VII (Sly syndrome)
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Review of designation
Documents related to this orphan designation evaluation
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: